A Bayesian joint bent-cable model for longitudinal measurements and survival time with heterogeneous random-effects distributions.

IF 1.2 4区 医学 Q4 PHARMACOLOGY & PHARMACY Journal of Biopharmaceutical Statistics Pub Date : 2025-01-20 DOI:10.1080/10543406.2025.2450321
Oludare Ariyo, Kehinde Olobatuyi, Taban Baghfalaki
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Abstract

Biomarkers are measured repeatedly in clinical studies until a pre-defined endpoint, such as death from certain causes, is reached. Such repeated measurements may present a dynamic process for understanding when to expect the study's endpoint. Joint modelling is often employed to handle such a model. Typically, shared random effects are assumed to be common to both the longitudinal component and the study's endpoint. These shared random effects usually assume homogeneous and follow a normal distribution. However, identifying homogeneous subgroups is important when the underlying population is heterogeneous. This issue has received little attention in the literature, particularly for multi-phase longitudinal responses. In this paper, we propose a joint modelling approach for longitudinal and survival models using a bent-cable mixed model for longitudinal measurements and a Weibull distribution for the survival component. We also incorporate a finite mixture of normal distribution assumptions to account for the unobserved heterogeneity in the shared random effects model. A Bayesian MCMC is developed for parameter estimation and inferences. The proposed method is evaluated using simulation studies and the Tehran Lipid and Glucose Study dataset.

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具有非均匀随机效应分布的纵向测量和存活时间的贝叶斯节点弯索模型。
在临床研究中,生物标志物被反复测量,直到达到预定的终点,如某些原因导致的死亡。这种重复的测量可能会呈现一个动态的过程,以便了解何时期望研究的终点。通常采用关节建模来处理这种模型。通常,假设共享随机效应对纵向成分和研究终点都是共同的。这些共有的随机效应通常假设是均匀的,并遵循正态分布。然而,当潜在人群是异质的时候,确定同质亚群是很重要的。这个问题在文献中很少受到关注,特别是对于多相纵向响应。在本文中,我们提出了纵向和生存模型的联合建模方法,使用纵向测量的弯曲-电缆混合模型和生存分量的威布尔分布。我们还纳入了正态分布假设的有限混合,以解释共享随机效应模型中未观察到的异质性。提出了一种用于参数估计和推理的贝叶斯MCMC。使用模拟研究和德黑兰脂质和葡萄糖研究数据集对所提出的方法进行了评估。
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来源期刊
Journal of Biopharmaceutical Statistics
Journal of Biopharmaceutical Statistics 医学-统计学与概率论
CiteScore
2.50
自引率
18.20%
发文量
71
审稿时长
6-12 weeks
期刊介绍: The Journal of Biopharmaceutical Statistics, a rapid publication journal, discusses quality applications of statistics in biopharmaceutical research and development. Now publishing six times per year, it includes expositions of statistical methodology with immediate applicability to biopharmaceutical research in the form of full-length and short manuscripts, review articles, selected/invited conference papers, short articles, and letters to the editor. Addressing timely and provocative topics important to the biostatistical profession, the journal covers: Drug, device, and biological research and development; Drug screening and drug design; Assessment of pharmacological activity; Pharmaceutical formulation and scale-up; Preclinical safety assessment; Bioavailability, bioequivalence, and pharmacokinetics; Phase, I, II, and III clinical development including complex innovative designs; Premarket approval assessment of clinical safety; Postmarketing surveillance; Big data and artificial intelligence and applications.
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